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Rigel Finalizes the Study Design of its Ongoing Phase 3 Clinical Trial of Fostamatinib in Warm Autoimmune Hemolytic Anemia
FDA agrees to the proposed primary efficacy endpoint and additional secondary endpoints SOUTH SAN FRANCISCO, Calif., Nov. 17, 2020 /PRNewswire/ -- Rigel
About this update from Rigel Pharmaceuticals, Inc.
[{"type":"text","content":"FDA agrees to the proposed primary efficacy endpoint and additional secondary endpoints\n\n\nSOUTH SAN FRANCISCO, Calif., Nov. 17, 2020 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on the final design of its FORWARD study, a pivotal Phase 3 clinical trial of fostamatinib disodium hexahydrate (fostamatinib) in warm autoimmune hemolytic anemia (AIHA). The FDA agreed to Rigel's proposed durable response measure for the primary efficacy endpoint as well as the inclusion of additional secondary endpoints. \nThe Phase 3 clinical trial is a randomized, double-blind, placebo-controlled study of approximately 90 patients with primary or secondary warm AIHA who have failed at least one prior treatment. The primary efficacy endpoint for the trial is a durable response defined as a hemoglobin level ≥ 10 g/dl with an increase from baseline of ≥ 2 g/dl on three consecutive available visits during the 24-week treatment period, with the response not being attributed to rescue therapy. This endpoint allows for missed visits due to the COVID-19 pandemic without impacting a durable outcome. As of November 5, the trial had enrolled 57 of the 90 patients targeted for enrollment and currently has over 90 clinical trial sites established across 22 countries. \n\"Our conversations with the FDA have enabled us to finalize the primary efficacy endpoint for the only ongoing Phase 3 trial in warm AIHA, a condition for which there is no approved therapy,\" said Raul Rodriguez, Rigel's president and CEO. \"We are over 60% of our enrollment target despite the headwinds drug development is facing due to COVID-19. We believe this progress is a result of the geographic diversity of our clinical sites, the ability of fostamatinib to be given orally, and the FDA's clinical trial guidance during the pandemic.\"\nFostamatinib is commercially available in the U.S. under the brand name TAVALISSE® (fostamatinib disodium hexahydrate) tablets, which is the first and only spleen tyrosine kinase (SYK) inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment. The FDA has granted TAVALISSE Orphan Drug designation for the treatment of patients with warm AIHA.\nAbout AIHAAu...